WO2018014041A3 - Composés et procédés de modulation de smn2 - Google Patents
Composés et procédés de modulation de smn2 Download PDFInfo
- Publication number
- WO2018014041A3 WO2018014041A3 PCT/US2017/042463 US2017042463W WO2018014041A3 WO 2018014041 A3 WO2018014041 A3 WO 2018014041A3 US 2017042463 W US2017042463 W US 2017042463W WO 2018014041 A3 WO2018014041 A3 WO 2018014041A3
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- compounds
- smn2
- methods
- modulation
- compositions
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 abstract 1
- 102100021947 Survival motor neuron protein Human genes 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 238000004519 manufacturing process Methods 0.000 abstract 1
- 208000002320 spinal muscular atrophy Diseases 0.000 abstract 1
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Genetics & Genomics (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurosurgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Dermatology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Pain & Pain Management (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Steroid Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Abstract
Priority Applications (24)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
BR112019000356-8A BR112019000356A2 (pt) | 2016-07-15 | 2017-07-17 | compostos e métodos para modulação de smn2 |
DK17828625.8T DK3484524T3 (da) | 2016-07-15 | 2017-07-17 | Forbindelser og fremgangsmåder til modulation af smn2 |
IL264216A IL264216B2 (en) | 2016-07-15 | 2017-07-17 | Compounds and Methods for Modulating SMN2 |
FIEP17828625.8T FI3484524T3 (fi) | 2016-07-15 | 2017-07-17 | YHDISTEITÄ JA MENETELMIÄ SMN2:n MODULOIMISEKSI |
SG11201900238UA SG11201900238UA (en) | 2016-07-15 | 2017-07-17 | Compounds and methods for modulation of smn2 |
JP2019500807A JP7197463B2 (ja) | 2016-07-15 | 2017-07-17 | Smn2の調節のための化合物及び方法 |
MX2019000619A MX2019000619A (es) | 2016-07-15 | 2017-07-17 | Compuestos y metodos para la modulacion de smn2. |
LTEPPCT/US2017/042463T LT3484524T (lt) | 2016-07-15 | 2017-07-17 | Junginiai ir būdai, skirti smn2 moduliavimui |
AU2017297622A AU2017297622A1 (en) | 2016-07-15 | 2017-07-17 | Compounds and methods for modulation of SMN2 |
PL17828625.8T PL3484524T3 (pl) | 2016-07-15 | 2017-07-17 | Związki i sposoby modulacji smn2 |
US16/310,766 US20190323006A1 (en) | 2016-07-15 | 2017-07-17 | Compounds and methods for modulation of smn2 |
RS20221176A RS63928B1 (sr) | 2016-07-15 | 2017-07-17 | Jedinjenja i metode za modulaciju smn2 |
EP22206076.6A EP4206213A1 (fr) | 2016-07-15 | 2017-07-17 | Composés et procédés de modulation de smn2 |
IL308256A IL308256A (en) | 2016-07-15 | 2017-07-17 | Compounds and Methods for Modulating SMN2 |
ES17828625T ES2935658T3 (es) | 2016-07-15 | 2017-07-17 | Compuestos y métodos para la modulación de SMN2 |
CN201780043645.5A CN109844115B (zh) | 2016-07-15 | 2017-07-17 | 用于调节smn2的化合物和方法 |
EP17828625.8A EP3484524B1 (fr) | 2016-07-15 | 2017-07-17 | Composés et procédés de modulation de smn2 |
CA3030864A CA3030864A1 (fr) | 2016-07-15 | 2017-07-17 | Composes et procedes de modulation de smn2 |
KR1020197004350A KR102556825B1 (ko) | 2016-07-15 | 2017-07-17 | Smn2 조정을 위한 조성물 및 방법 |
HRP20221378TT HRP20221378T1 (hr) | 2016-07-15 | 2017-07-17 | Spojevi i postupci za modulaciju smn2 |
SI201731272T SI3484524T1 (sl) | 2016-07-15 | 2017-07-17 | Spojine in postopki za modulacijo SMN2 |
CONC2019/0001236A CO2019001236A2 (es) | 2016-07-15 | 2019-02-12 | Compuestos y métodos para la modulación de smn2 |
US17/127,866 US20220073914A1 (en) | 2016-07-15 | 2020-12-18 | Compounds and methods for modulation of smn2 |
JP2022199992A JP2023027285A (ja) | 2016-07-15 | 2022-12-15 | Smn2の調節のための化合物及び方法 |
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662363195P | 2016-07-15 | 2016-07-15 | |
US62/363,195 | 2016-07-15 |
Related Child Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
US16/310,766 A-371-Of-International US20190323006A1 (en) | 2016-07-15 | 2017-07-17 | Compounds and methods for modulation of smn2 |
US17/127,866 Continuation US20220073914A1 (en) | 2016-07-15 | 2020-12-18 | Compounds and methods for modulation of smn2 |
Publications (2)
Publication Number | Publication Date |
---|---|
WO2018014041A2 WO2018014041A2 (fr) | 2018-01-18 |
WO2018014041A3 true WO2018014041A3 (fr) | 2019-04-18 |
Family
ID=60952211
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2017/042463 WO2018014041A2 (fr) | 2016-07-15 | 2017-07-17 | Composés et procédés de modulation de smn2 |
Country Status (25)
Country | Link |
---|---|
US (2) | US20190323006A1 (fr) |
EP (2) | EP3484524B1 (fr) |
JP (2) | JP7197463B2 (fr) |
KR (1) | KR102556825B1 (fr) |
CN (1) | CN109844115B (fr) |
AU (1) | AU2017297622A1 (fr) |
BR (1) | BR112019000356A2 (fr) |
CA (1) | CA3030864A1 (fr) |
CL (2) | CL2019000115A1 (fr) |
CO (1) | CO2019001236A2 (fr) |
DK (1) | DK3484524T3 (fr) |
ES (1) | ES2935658T3 (fr) |
FI (1) | FI3484524T3 (fr) |
HR (1) | HRP20221378T1 (fr) |
HU (1) | HUE060831T2 (fr) |
IL (2) | IL264216B2 (fr) |
LT (1) | LT3484524T (fr) |
MX (1) | MX2019000619A (fr) |
PE (1) | PE20190513A1 (fr) |
PL (1) | PL3484524T3 (fr) |
PT (1) | PT3484524T (fr) |
RS (1) | RS63928B1 (fr) |
SG (1) | SG11201900238UA (fr) |
SI (1) | SI3484524T1 (fr) |
WO (1) | WO2018014041A2 (fr) |
Families Citing this family (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2014169243A2 (fr) * | 2013-04-12 | 2014-10-16 | The Curators Of The University Of Missouri | Compositions antisens de l'élément 1 de smn2 et leurs procédés et utilisations |
US10053465B2 (en) | 2015-08-26 | 2018-08-21 | Incyte Corporation | Pyrrolopyrimidine derivatives as TAM inhibitors |
SI3436461T1 (sl) | 2016-03-28 | 2024-03-29 | Incyte Corporation | Pirolotriazinske spojine kot tam-inhibitorji |
RS62872B1 (sr) | 2017-09-27 | 2022-02-28 | Incyte Corp | Soli derivata pirrolotriazina korisne kao tam inhibitori |
CN117771250A (zh) | 2018-06-29 | 2024-03-29 | 因赛特公司 | Axl/mer抑制剂的制剂 |
EP4013387A4 (fr) * | 2019-08-15 | 2023-09-27 | Biogen MA Inc. | Polythérapie pour atrophie musculaire spinale |
WO2021054370A1 (fr) * | 2019-09-18 | 2021-03-25 | 国立大学法人東京医科歯科大学 | Complexe d'acide nucléique |
BR112022016238A2 (pt) * | 2020-02-28 | 2022-10-11 | Ionis Pharmaceuticals Inc | Compostos e métodos para modular smn2 |
FR3128873A1 (fr) | 2021-11-10 | 2023-05-12 | Biophytis | Phytoecdysones et/ou dérivés de 20-hydroxyecdysone en combinaison avec un principe actif visant à restaurer l’expression SMN pour leur utilisation dans le traitement de l’amyotrophie spinale |
Citations (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20130289092A1 (en) * | 2010-06-15 | 2013-10-31 | Cold Spring Harbor Laboratory | Compounds and methods for modulating interaction between proteins and target nucleic acids |
US20160068845A1 (en) * | 2010-07-19 | 2016-03-10 | University Of Rochester | Modulation of dystrophia myotonica-protein kinase (dmpk) expression |
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